TY - JOUR
T1 - The early economic evaluation of novel biomarkers to accelerate their translation into clinical applications
AU - Graaf, Gimon
AU - Postmus, Douwe
AU - Westerink, Jan
AU - Buskens, Erik
N1 - Funding Information:
This research was performed within the framework of CTMM, the Center for Translational Molecular Medicine http://www.ctmm.nl, project PREDICCt grant 01C-104, and supported by The Netherlands Heart Foundation, Dutch Diabetes Research Foundation and Dutch Kidney Foundation. The funding bodies had no influence on the design of the study, the collection, analysis, and interpretation of data, or the writing of the manuscript.
Publisher Copyright:
© 2018 The Author(s)..
PY - 2018/6/18
Y1 - 2018/6/18
N2 - Background: Translating prognostic and diagnostic biomarker candidates into clinical applications takes time, is very costly, and many candidates fail. It is therefore crucial to be able to select those biomarker candidates that have the highest chance of successfully being adopted in the clinic. This requires an early estimate of the potential clinical impact and commercial value. In this paper, we aim to demonstratively evaluate a set of novel biomarkers in terms of clinical impact and commercial value, using occurrence of cardiovascular disease (CVD) in type-2 diabetes (DM2) patients as a case study. Methods: We defined a clinical application for the novel biomarkers, and subsequently used data from a large cohort study in The Netherlands in a modeling exercise to assess the potential clinical impact and headroom for the biomarkers. Results: The most likely application of the biomarkers would be to identify DM2 patients with a low CVD risk and subsequently withhold statin treatment. As a result, one additional CVD event in every 75 patients may be expected. The expected downstream savings resulted in a headroom for a point-of-care device ranging from €119.09 at a willingness to accept of €0 for one additional CVD event, to €0 at a willingness to accept of €15,614 or more. Conclusion: It is feasible to evaluate novel biomarkers on outcomes directly relevant to technological development and clinical adoption. Importantly, this may be attained at the same point in time and using the same data as used for the evaluation of association with disease and predictive power.
AB - Background: Translating prognostic and diagnostic biomarker candidates into clinical applications takes time, is very costly, and many candidates fail. It is therefore crucial to be able to select those biomarker candidates that have the highest chance of successfully being adopted in the clinic. This requires an early estimate of the potential clinical impact and commercial value. In this paper, we aim to demonstratively evaluate a set of novel biomarkers in terms of clinical impact and commercial value, using occurrence of cardiovascular disease (CVD) in type-2 diabetes (DM2) patients as a case study. Methods: We defined a clinical application for the novel biomarkers, and subsequently used data from a large cohort study in The Netherlands in a modeling exercise to assess the potential clinical impact and headroom for the biomarkers. Results: The most likely application of the biomarkers would be to identify DM2 patients with a low CVD risk and subsequently withhold statin treatment. As a result, one additional CVD event in every 75 patients may be expected. The expected downstream savings resulted in a headroom for a point-of-care device ranging from €119.09 at a willingness to accept of €0 for one additional CVD event, to €0 at a willingness to accept of €15,614 or more. Conclusion: It is feasible to evaluate novel biomarkers on outcomes directly relevant to technological development and clinical adoption. Importantly, this may be attained at the same point in time and using the same data as used for the evaluation of association with disease and predictive power.
KW - Biomarkers
KW - Cardiovascular disease risk
KW - Early health technology assessment
KW - Headroom analysis
KW - Translational research
UR - http://www.scopus.com/inward/record.url?scp=85048723426&partnerID=8YFLogxK
U2 - 10.1186/s12962-018-0105-z
DO - 10.1186/s12962-018-0105-z
M3 - Article
AN - SCOPUS:85048723426
SN - 1478-7547
VL - 16
JO - Cost Effectiveness and Resource Allocation
JF - Cost Effectiveness and Resource Allocation
IS - 1
M1 - 23
ER -